Safety and Efficacy of Mesenchymal Stromal Cells Derived from Adipose Tissue of Non-related Donors in Patients with The Butterfly Disease: Clinical Trial Phase I/II

Phase 1

• Conditions: Epidermolysis bullosa congenita; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2020-002936-55-CZ
• Lead Sponsor: Masarykova univerzita

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-08-03
• Last Update Posted: 4 April 2022

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

1) Age > 1 year (incl.)
2) Clinical diagnosis of hereditary EB supported by histological diagnosis (electron microscopy, immunofluorescence antigen mapping) and DNA analysis
3) The extent of the disease is at least 10% of the body surface
4) Participants in a clinical trial of childbearing potential must agree to the use of prescribed contraceptive methods for the duration of the clinical study and for at least 6 months following the last dose of the study medication:
a) Women - Proper use of a highly reliable method of contraception, ie combined hormonal contraception (in oral, vaginal or transdermal dosage form), gestagen hormonal contraception associated with ovulation inhibition (in oral or injectable dosage form), non-hormonal intrauterine device or intrauterine device releasing hormones, or the presence of bilateral tubal occlusion, a partner's vasectomy, or adherence to sexual abstinence as part of the patient's normal lifestyle.
b) Men - Adherence to sexual abstinence or the use of an adequate contraceptive method (ie condom) in case of sexual intercourse.
5) Willingness and ability to adhere to restrictions in the care of study lesions and to adhere to the schedule of visits and examinations during the course of the study.
Are the trial subjects under 18? yes
Number of subjects for this age range: 11
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 4
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1) Systemic infection or sepsis 7 days before MSC administration
2) Treatment with blood transfusion (erythrocyte concentrate or whole blood transfusion) 7 days before MSC administration
3) Clinical signs of infection in the study lesions 7 days before MSC administration
4) Inability to tolerate repeated skin injections (especially in pediatric patients)
5) History of basal cell or squamous cell carcinoma of the skin in the last 5 years
6) History of other malignancies of any type at any time during life
7) Severe lung disease that requires home oxygenation
8) Severe kidney or liver disease
9) Dilatation cardiomyopathy
10) Life expectancy less than 90 days
11) Severe immune response to allogeneic human cells
12) Other severe somatic or psychiatric illnesses that are not adequately controlled and, in the opinion of the investigator, would not allow the study protocol to be followed
13) Previous administration of a medicinal product in a clinical trial in the 30 days or 5 elimination half-lives (whichever is longer) before the enrolment to this trial (applies to clinical investigations of medical devices, as well)
14) Pregnancy or breastfeeding
15) Allergy/hypersensitivity to any component of Hypotermosol solution

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified